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Table 8 Statistical analysis of the pivotal studies (n = 108)

From: Clinical evidence for orphan medicinal products-a cause for concern?

  Number of pivotal studies (%)
Appropriate statistical analysis
 Yes 48 (44.4%)
 No 41 (38.0%)
 Don’t know (too few details) 19 (17.6%)
Summary statistics at baseline
 Yes 71 (65.7%)
 With statistical testing 7 (6.5%)
Summary statistics at outcome
 No 5 (4.6%)
 Yes, for all endpoints 44 (40.7%)
      Statistically tested 14 (13.0%)
      Not statistically tested 30 (27.8%)
 Yes, partially 45 (41.7%)
      Statistically tested 31 (28.7%)
      Not statistically tested 14 (13.0%)
 Yes, only primary 14 (13.0%)
      Statistically tested 10 (9.3%)
      Not statistically tested 4 (3.7%)
Intention-to-treat analysis
 Yes 74 (68.5%)
 No 8 (7.4%)
 Not applicable / Not reported 26 (24.0%)
Reported with respect to patients lost to follow-up
 Reasons for loss to follow-up 70 (64.8%)
 Characteristics 2 (1.9%)
 Not applicable 8 (7.4%)